Archives: Agenda

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• Considering ways to boost recruitment through investigator meetings and recruitment workshops
• Communicating effectively and working together with sites to assist in recruitment
• Sharing recruitment challenges and successes from a rare disease trial that started during Covid-19 pandemic

• Clinical study design and key outcomes in sporadic ALS patients
• Mechanistic insights: targeting mitochondrial dysfunction as a therapeutic strategy
• Lessons learned and implications for future rare disease trials

• Thinking ahead and moving beyond protocol approval to consider downstream data use, publication, and medical education needs
• Maximizing the value of limited datasets so every data point supports meaningful insights and avoids missed opportunities.
• Bridging clinical, medical affairs and data strategy to improving early cross-functional collaboration to prevent limitations in data dissemination later

• Sharing insights on how to use AI to bring drugs to rare disease patients
• Running through step by step processes including pre IND, FDA work, organising and conducting trial
• Discussing challenges, successes and real-world experience

• Creating vendor selection criteria and processes
• Establishing shared goals and KPIs to foster trust and alignment
• Managing expectations on timelines, budgets and deliverables
• Evaluating vendor capability in finding and supporting rare disease patients
• Monitoring vendor performance, governance and communication plans